CLAG+Ven挽救治疗复发难治急性髓系白血病的疗效及安全性:一项单中心单臂临床研究  

Clinical efficacy and safety of CLAG+Ven as salvage therapy for relapsed/refractory acute myeloid leukemia:A single-center,single-arm clinical study

作　　者：姚足容 尹昭 陈丹丹[1] 郭俊良 史鹏程[1] 徐丹[1] 于嗣俭 叶洁瑜[1] 范志平[1] 许娜[1] 黄芬[1] 林韧[1] 金华 宣丽[1] 刘启发[1] 张钰[1,2] 余国攀 YAO Zurong;YIN Zhao;CHEN Dandan;GUO Junliang;SHI Pengcheng;XU Dan;YU Sijian;YE Jieyu;FAN Zhiping;XU Na;HUANG Fen;LIN Ren;JIN Hua;XUAN Li;LIU Qifa;ZHANG Yu;YU Guopan(Department of Hematology,Nanfang Hospital,Southern Medical University,Guangzhou,510515,China;Guangdong Provincial Clinical Research Center for Hematological Diseases)

机构地区：[1]南方医科大学南方医院血液科,广州510515 [2]广东省血液病临床医学研究中心

出　　处：《临床血液学杂志》2025年第3期184-190,共7页Journal of Clinical Hematology

基　　金：国家自然科学基金(No:82270178);广东省自然科学基金(No:2023A1515010111)。

摘　　要：目的:研究维奈克拉(Ven)联合CLAG方案挽救性治疗复发难治急性髓系白血病(RR-AML)的疗效和安全性。方法:开展单中心单臂临床研究使用CLAG+Ven挽救治疗RR-AML,主要终点为复合缓解(CRc)率,次要终点为总反应率(ORR)、可检测残留病(MRD)、无事件生存(EFS)、总生存(OS)和安全性分析。结果:2021年7月—2024年12月共有47例RR-AML患者接受1个疗程CLAG+Ven治疗并完成疗效和安全性评估,中位年龄42(18~65)岁,37例(78.7%)患者获得治疗反应,其中32例(68.1%)达到CRc,21例(65.6%)获得MRD阴性CRc。欧洲白血病网络低、中、高危组或细胞遗传学非高危和高危组患者的CRc率均差异无统计学意义。亚组分析显示既往去甲基化药物(HMA)暴露对患者获得CRc不利。中位随访13.0(8.7~17.3)个月,中位OS、中位EFS均未达到,1年OS率为72%(95%CI:63.84%~80.52%)。CLAG+Ven治疗患者中性粒细胞恢复至>1×10^(9)/L的中位时间为18(7~58)d,30d和60d死亡率分别为2.1%和6.4%。结论:CLAG+Ven方案挽救治疗RR-AML有较好疗效,耐受性良好。Objective:To investigate the clinical efficacy and safety of the CLAG(Cladribine,Cytarabine,and G-CSF)plus Venetoclax(Ven)regimen as salvage therapy for relapsed/refractory acute myeloid leukemia(RRAML).Methods:A single-center,single-arm study was performed to evaluate the clinical efficacy and safety of CLAG+Ven regimen in patients with RR-AML.The primary endpoint was the composite complete remission(CRc)rate.Secondary endpoints included the overall response rate(ORR),measurable residual disease(MRD),event-free survival(EFS),overall survival(OS),and safety analysis.Results:From July 2021to December 2024,a total of 47patients with RR-AML were included,who have received one course of CLAG+Ven therapy and completed response evaluation,with a median age of 42(range 18-65)years.Thirty-seven patients(78.7%)acquired response,including 32(68.1%)with CRc and 21(65.6%)with MRD-negative CRc.There were no statistical differences in the CRc rates among the different risk groups based on ELN stratification and cytogenetic classification.Subgroup analysis showed that prior exposure to hypomethylating agents(HMAs)was associated with a poor CRc.With a median follow-up of 13(8.7-17.3)months,median OS and EFS were not reached,and the 1-year OS rate was 72%(95%CI:63.84%-80.52%).The median time for neutrophil recovery(Neu>1×10^(9)/L)was 18(range 7-58)days,with the 30-and 60-day mortality rates of 2.1%and 6.4%,respectively.Conclusion:The CLAG+Ven regimen as salvage therapy demonstrated a promising efficacy in patients with RR-AML and was·184·well-tolerated.

关 键 词：维奈克拉 CLAG 难治复发 挽救治疗 急性髓系白血病 

分 类 号：R733.7[医药卫生—肿瘤]